Hematopoietic Stem Cell Transplantation in Patients with Hemoglobinopathies

Yeşilipek, M. Akif

doi:10.1080/03630269.2020.1832516

Cited by 9 publications

(16 citation statements)

References 69 publications

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“…8,25 Protocol 26, the conditioning regimen recommended by the Pesaro group for class 3 patients, was used. 1 All patients who underwent allogeneic HSCT received prophylactic antimicrobial prophylaxis.…”

Section: Methodsmentioning

confidence: 99%

“…Approximately 300,000 babies are born with β-TM each year. 1 In transfusion-dependent thalassemia patients, the excess iron that accumulates in the body causes organ damage, leading to severe morbidity and mortality. Standard treatment for β-TM involves regular blood transfusion, iron overload reduction, and splenectomy if necessary.…”

mentioning

confidence: 99%

“…5 With age, organ dysfunction caused by iron overload results in a higher risk of transplant complications and mortality; therefore, fewer transplantations are performed in adults. 1 Current experience shows that overall survival after HSCT is over 90%, and thalassemia-free survival is around 85%. 2,4,5,8 Clinical experience with HSCT in thalassemia patients is steadily growing.…”

mentioning

confidence: 99%

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Efficacy and Safety of Iron Chelation Therapy After Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric Thalassemia Patients: A Retrospective Observational Study

Küpesiz

Sivrice²,

Akinel³

et al. 2021

Journal of Pediatric Hematology/Oncology

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Background: Studies on the increased body iron load in patients with thalassemia major have thoroughly demonstrated the problems caused by iron overload. In patients who undergo hematopoietic stem cell transplantation (HSCT) as curative therapy, iron overload continues long after transplantation. There are few pediatric studies on chelation therapy in the posttransplant period. In this study, we present the outcomes of our patients who received posttransplant oral chelation therapy.Patients and Methods: This retrospective observational study evaluated the outcomes of pediatric patients with thalassemia major who used oral chelation therapy after allogeneic HSCT at the Akdeniz University Pediatric Bone Marrow Unit between January 2008 and October 2019.Results: Deferasirox therapy was initiated in 58 pediatric patients who underwent HSCT for thalassemia. Pretreatment mean serum ferritin was 2166 ± 1038 ng/mL. Treatment was initiated at a mean of 12 ± 6.7 months after transplantation and continued for a mean of 15.7 ± 11.5 months. At treatment discontinuation, the mean serum ferritin was 693 ± 405 ng/mL and the mean reduction was −1472.75 ± 1121.09 ng/mL (P < 0.001 vs. posttreatment). Serum ferritin was below 500 ng/mL in 52% of the patients at treatment discontinuation. Manageable side effects such as nausea, vomiting, liver enzyme elevation, and proteinuria were observed in 17% of the patients, while one patient developed ototoxicity.Conclusions: Deferasirox therapy effectively reduces iron overload in the posttransplant period. Studies evaluating the effects of early treatment on the graft may help to establish guidelines for posttransplant chelation therapy. Clear guidelines are needed regarding when to initiate and discontinue treatment.

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Section: Methodsmentioning

confidence: 99%

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confidence: 99%

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confidence: 99%

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Efficacy and Safety of Iron Chelation Therapy After Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric Thalassemia Patients: A Retrospective Observational Study

Küpesiz

Sivrice²,

Akinel³

et al. 2021

Journal of Pediatric Hematology/Oncology

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“…Management of patients often includes regular blood transfusion throughout life as well as iron chelation therapy 7 . Regarding treatment, haematopoietic stem cell transplantation (HSCT) is one option for patients with severe hemoglobinopathies 8 . However, this is hampered by the availability of a suitable donor.…”

Section: Hemoglobinopathiesmentioning

confidence: 99%

“…7 Regarding treatment, haematopoietic stem cell transplantation (HSCT) is one option for patients with severe hemoglobinopathies. 8 However, this is hampered by the availability of a suitable donor. Several clinical trials and applications of gene therapy and editing for hemoglobinopathies have been reported.…”

Section: Hemoglobinopathiesmentioning

confidence: 99%

Hemoglobinopathies and preimplantation diagnostics

Mamas

Kakourou

Vrettou

et al. 2022

Int J Lab Hematology

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Hemoglobinopathies constitute some of the most common inherited disorders worldwide. Manifestations are very severe, patient management is difficult and treatment is not easily accessible. Preimplantation genetic testing for monogenic disorders (PGT-M) is a valuable reproductive option for hemoglobinopathy carrier-couples as it precludes the initiation of an affected pregnancy. PGT-M is performed on embryos generated by assisted reproductive technologies and only those found to be free of the monogenic disorder are transferred to the uterus. PGT-M has been applied for 30 years now and β-thalassemia is one of the most common indications. PGT may also be applied for human leukocyte antigen typing to identify embryos that are unaffected and also compatible with an affected sibling in need of hemopoietic stem cell transplantation. PGT-M protocols have evolved from PCR amplification-based, where a small number of loci were analysed, to whole genome amplification-based, the latter increasing diagnostic accuracy, enabling the development of more generic strategies and facilitating multiple diagnoses in one embryo. Currently, numerous PGT-M cycles are performed for the simultaneous diagnosis of hemoglobinopathies and screening for chromosomal abnormalities in the embryo in an attempt to further improve success rates and increase deliveries of unaffected babies.

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